Drug regulators in the United States have finally approved an old drug that has never been available in America for the treatment of a rare disease known to afflict only about 15,000 Americans, annually. Before now, this drug—known as deflazacort—has been available to patients who suffer from a quite devastating and generally fatal disease, Duchenne muscular dystrophy. Previously, then, families in need of the drug could import it for roughly $1,200 a year.
While the condition is rare, the drug is important, and that might explain why it has now been approved for sale in the United States, though, its new price tag might make you wish it hadn’t.
The new, American, list price for this drug is $89,000 a year. Now, the company that makes the drug—Marathon Pharmaceuticals—wants to make sure that you know that after rebates and discounts, you can get the treatment for $54,000, according to CFO Babar Ghias.
As you might expect, the industry—and critics all over—are up in arms. Harvard Medical School associate professor of medicine, Aaron Kesselheim, for example, comments, “It seems like it’s yet another example of gaming the system. How many examples of this do we have to see before we can start to rethink the priority review voucher as a means of incentivizing innovation? This also seems to be another example of gaming the Orphan Drug Act, which was intended to try and encourage research into new therapeutic entities for people who have rare diseases — and it doesn’t seem like this is that.”
Ghias returns to advise that the cost of the drug—under the brand name Emflaza—will be a “zero to low out-of pocket expense” to all patients. Still, the price jump is alarming; and, again, Ghias defends that high drug prices are a form of protection that insulates companies from harsh criticism to ultimately deliver the drug to the families with dying children who need it most.
To that, of course, Kesselheim responds: “Instead of making the price at a level that is reasonable for patients, they make it a very high price and offer this pathway that patients may not qualify for, they may not know about, there may be limitations on it. So it’s a marketing move and not really a public health solution.”